Latest news with #MDS-UPDRS
Yahoo
21-05-2025
- Business
- Yahoo
ABLi Therapeutics Announces Details of the End of Phase 2 Meeting with FDA for Evaluation of Risvodetinib as a Treatment for Parkinson's Disease
Agency supports primary and novel secondary and exploratory endpoints proposed for Phase 3 Trial(s) Opportunity to utilize time-to-delay of levodopa/carbidopa initiation as an endpoint to measure trial success, which Company believes is a direct measure of disease-modification ATLANTA and BOSTON, May 21, 2025 (GLOBE NEWSWIRE) -- ABLi Therapeutics ('ABLi'), a biotechnology company developing therapeutics to address diseases that arise from activation of Abelson Tyrosine Kinases (c-Abl kinases), completed an End of Phase 2 (EOP2) meeting with the Food and Drug Administration (FDA) to discuss outcomes of the 201 Trial in untreated Parkinson's disease and to review plans for the Phase 3 program. The meeting's primary purpose was to discuss endpoints to be used in a Phase 3 program and to define what tangential clinical and pre-clinical experiments may be necessary for a potential filing of a New Drug Application (NDA.) The Company presented data showing that the 201 Trial reached nominal significance for the Movement Disorder Society revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and for the Schwab & England Activities of Daily Life (SEADL) scale in a 12 week treatment, which were consistent with the FDA's preference for evaluating patient quality of life as a primary assessment of clinical benefit. The Company proposed to use the change in the MDS-UPDRS Part 2 as the primary outcome measure for its planned Phase 3 trial, consistent with the Agency's view of the most clinically meaningful outcome for patients. The Agency encouraged continued use of Patient and Clinical Severity scales as was done in the Phase 2 trial to complement MDS-UPDRS and SEADL measures. The Company further proposed a novel endpoint to determine if risvodetinib lengthens the time before levodopa/carbidopa treatment is initiated. In the Company's view, lengthening this time from the beginning of the Phase 3 trial relative to the placebo-treated controls represents a direct measure of modifying the course of Parkinson's disease. The FDA agreed with the use of this approach and encouraged a follow-up meeting to align on the clinical standard to be used to determine when a participant should go onto levodopa/carbidopa treatment. The meeting simplified the requirement included in the Phase 2 trial for thorough monitoring of vision, and reduced the frequency of thorough monitoring to a six-month interval until one year dosing data is reviewed by the Agency. To date, no risvodetinib-related impact on vision has been observed for any participant administered any dose of risvodetinib once daily for up to 12 weeks. The Agency encouraged the Company to continue its evaluation of skin biopsy and measures in other biological fluids before determining if the Company's approach using skin biopsy could become a prognostic marker of treatment success. The Company views the quantitative measure of alpha-synuclein clearance as a supportive measure alongside clinical outcome assessments because it is now well-established that all forms of Parkinsonism arise from neuronal alpha-synuclein deposition in a variety of aggregated and fibrillar forms that may be characteristic of different types of Parkinson's disease. Risvodetinib is the first Parkinson's treatment that reduced pathological alpha-synuclein in the context of a clinical benefit. About Risvodetinib (ABLi-148009)Risvodetinib is a potent, selective small-molecule inhibitor of the non-receptor c-Abl kinases, designed for once-daily oral use that targets the underlying biological mechanisms driving Parkinson's disease initiation and progression. Risvodetinib is believed to be a disease-modifying therapy that halts disease progression and reverses the functional loss arising from Parkinson's disease inside and outside of the brain. All marketed therapeutic approaches to treat Parkinson's help manage the symptoms of the disease, but there are currently no available treatments to slow or stop the disease's relentless progression. Recently, risvodetinib was the first monotherapy to improve patient quality of life in a randomized, placebo-controlled clinical trial (NCT NCT05424276) and simultaneously reduced the underlying disease pathology in untreated Parkinson's disease. Risvodetinib currently has intellectual property protection beyond 2036. About ABLi TherapeuticsABLi Therapeutics ('ABLi') applies innovative medicinal chemistry and a deep understanding of disease biology to develop small molecule therapeutics that target the cause of diseases that arise from activation or dysfunction of the Abelson Tyrosine Kinases (c-Abl). Leveraging its expertise in drug design, ABLi utilizes clinically validated data of kinase inhibitors to design and develop novel product candidates with enhanced penetration into the brain, greater potency and target selectivity, and improved safety to treat diseases in which Abl kinase activation or dysfunction is implicated. The Company's primary focus is on developing therapeutics for the treatment of neurodegenerative diseases like Parkinson's disease and the Parkinson's-related neurodegenerative diseases Multiple System Atrophy and Dementia with Lewy Body that are all associated with Abl kinase activation or dysfunction. For more information visit or follow us on LinkedIn. Contacts: For ABLi TherapeuticsMilton H Werner PhDChairman & CEOinfo@ Investor/MediaMike MoyerManaging Director – LifeSci Advisorsmmoyer@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
22-04-2025
- Health
- Yahoo
Stem-cell therapy for Parkinson's shows 'strong promise' in relieving symptoms
With around one million people living with Parkinson's disease in the U.S. — and 90,000 getting new diagnoses each year — the race is on for a cure. Researchers at Memorial Sloan Kettering Cancer Center (MSK) have announced progress on that front — they have developed a new therapy that uses stem cells to treat advanced Parkinson's. In the phase 1 trial, the researchers used donated stem cells (taken from early-stage embryos) to create nerve cells (neurons) and transplanted them into the brains of 12 Parkinson's patients, according to a press release from MSK. New Drug For Parkinson's Shown To Be Effective In Clinical Trials: 'Very Encouraged' Once the cells are injected, they produce dopamine, a hormone in the brain that helps with movement and coordination. (One of the hallmarks of Parkinson's is low levels of dopamine, which causes the typical symptoms of tremors, stiffness, balance issues and difficulty walking.) Read On The Fox News App After 18 months, the injected cells had "taken hold in the brain with no serious side effects," the researchers reported. Based on the MDS-UPDRS — a ratings scale for symptoms developed by the International Parkinson and Movement Disorder Society — the participants experienced "noticeable improvements," especially the group that received a higher dose. "Neurologists say things usually get a little bit worse every year with this disease, meaning the score goes up by a few points," study co-author Lorenz Studer, MD, director of MSK's Center for Stem Cell Biology, said in the press release. "In our study, not only did the score not get worse, it dropped by more than 20 points in the high-dose group." On average, patients in the high-dose group reported 2.7 hours of additional "on time" — indicating periods of normal functioning with minimal symptoms — "a result that could be quite meaningful for their everyday life," Studer noted. Given the success of the phase 1 trial, the U.S. Food and Drug Administration (FDA) has granted approval for the researchers to go straight to a phase 3 clinical trial in a much larger patient group — around 100 people — which will take place in the first half of 2025. The findings were published in the journal Nature. Parkinson's Cases Could Double Globally By 2050, Study Reveals "The study showed that developing specific nerve cells from human embryonic stem cells in the lab, then injecting them in the brain of people with Parkinson's disease, is safe and holds significant promise as a possible future treatment," lead study author Viviane Tabar, MD, chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center in New York City, told Fox News Digital. "The findings were rewarding, as this work is more than a dozen years in the making." Dr. Mary Ann Picone, medical director of the MS Center at Holy Name Medical Center in Teaneck, New Jersey, said that cells for the treatment of Parkinson's disease could offer potential for not only slowing disability, but also stopping progression and bringing about improvement in motor function. "Although there is risk involved in the immune suppression necessary before the stem cell implantation and the procedure itself, it would be a major step forward in replacing the dopaminergic neurons lost in the disease," Picone, who was not involved in the study, told Fox News Digital. Levodopa, currently the first-line treatment for Parkinson's, is limited in that patients need greater dosage amounts as time goes on, according to Picone — "and regulating off and on periods of either stiffness or dyskinesias (uncontrolled muscle movements) becomes more difficult." Dr. Ann Murray, director of movement disorders at WVU Rockefeller Neuroscience Institute in West Virginia, referred to the study as "unbelievably exciting" for Parkinson's patients. "Although the goal of this particular research project was to ensure safety, getting that significant clinical improvement in the UPDRS is absolutely groundbreaking," Murray told Fox News Digital. (She also was not involved in the study.) Click Here To Sign Up For Our Health Newsletter "This is just the first step in getting this type of therapy approved for patients suffering from Parkinson's disease, but this is an amazing first step for the potential benefits of stem cell brain therapy." There were some limitations associated with the study, Tabar noted. "This is a small study designed to show safety — it is critical to conduct a larger, well-controlled study to prove that the treatment indeed works, otherwise referred to as a Phase 3 'efficacy' study," she said. These early findings, however, are "suggestive of a strong promise." "I think we can finally say that stem cells, when derived and differentiated properly, hold great promise to repair the brain in Parkinson's and potentially in other conditions someday," Tabar said. For more Health articles, visit The cell therapy was developed at MSK and was licensed to BlueRock Therapeutics in Massachusetts, which funded the article source: Stem-cell therapy for Parkinson's shows 'strong promise' in relieving symptoms
Fox News
22-04-2025
- Health
- Fox News
Stem-cell therapy for Parkinson's shows 'strong promise' in relieving symptoms
With around one million people living with Parkinson's disease in the U.S. — and 90,000 getting new diagnoses each year — the race is on for a cure. Researchers at Memorial Sloan Kettering Cancer Center (MSK) have announced progress on that front — they have developed a new therapy that uses stem cells to treat advanced Parkinson's. In the phase 1 trial, the researchers used donated stem cells (taken from early-stage embryos) to create nerve cells (neurons) and transplanted them into the brains of 12 Parkinson's patients, according to a press release from MSK. Once the cells are injected, they produce dopamine, a hormone in the brain that helps with movement and coordination. (One of the hallmarks of Parkinson's is low levels of dopamine, which causes the typical symptoms of tremors, stiffness, balance issues and difficulty walking.) After 18 months, the injected cells had "taken hold in the brain with no serious side effects," the researchers reported. Based on the MDS-UPDRS — a ratings scale for symptoms developed by the International Parkinson and Movement Disorder Society — the participants experienced "noticeable improvements," especially the group that received a higher dose. Patients in the high-dose group reported 2.7 hours of additional "on time" each day. "Neurologists say things usually get a little bit worse every year with this disease, meaning the score goes up by a few points," study co-author Lorenz Studer, MD, director of MSK's Center for Stem Cell Biology, said in the press release. "In our study, not only did the score not get worse, it dropped by more than 20 points in the high-dose group." On average, patients in the high-dose group reported 2.7 hours of additional "on time" — indicating periods of normal functioning with minimal symptoms — "a result that could be quite meaningful for their everyday life," Studer noted. Given the success of the phase 1 trial, the U.S. Food and Drug Administration (FDA) has granted approval for the researchers to go straight to a phase 3 clinical trial in a much larger patient group — around 100 people — which will take place in the first half of 2025. The findings were published in the journal Nature. "The study showed that developing specific nerve cells from human embryonic stem cells in the lab, then injecting them in the brain of people with Parkinson's disease, is safe and holds significant promise as a possible future treatment," lead study author Viviane Tabar, MD, chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center in New York City, told Fox News Digital. "The findings were rewarding, as this work is more than a dozen years in the making." Dr. Mary Ann Picone, medical director of the MS Center at Holy Name Medical Center in Teaneck, New Jersey, said that cells for the treatment of Parkinson's disease could offer potential for not only slowing disability, but also stopping progression and bringing about improvement in motor function. "Although there is risk involved in the immune suppression necessary before the stem cell implantation and the procedure itself, it would be a major step forward in replacing the dopaminergic neurons lost in the disease," Picone, who was not involved in the study, told Fox News Digital. Levodopa, currently the first-line treatment for Parkinson's, is limited in that patients need greater dosage amounts as time goes on, according to Picone — "and regulating off and on periods of either stiffness or dyskinesias (uncontrolled muscle movements) becomes more difficult." Dr. Ann Murray, director of movement disorders at WVU Rockefeller Neuroscience Institute in West Virginia, referred to the study as "unbelievably exciting" for Parkinson's patients. "Although the goal of this particular research project was to ensure safety, getting that significant clinical improvement in the UPDRS is absolutely groundbreaking," Murray told Fox News Digital. (She also was not involved in the study.) "This is just the first step in getting this type of therapy approved for patients suffering from Parkinson's disease, but this is an amazing first step for the potential benefits of stem cell brain therapy." There were some limitations associated with the study, Tabar noted. "This is a small study designed to show safety — it is critical to conduct a larger, well-controlled study to prove that the treatment indeed works, otherwise referred to as a Phase 3 'efficacy' study," she said. "This is an amazing first step for the potential benefits of stem cell brain therapy." These early findings, however, are "suggestive of a strong promise." "I think we can finally say that stem cells, when derived and differentiated properly, hold great promise to repair the brain in Parkinson's and potentially in other conditions someday," Tabar said. The cell therapy was developed at MSK and was licensed to BlueRock Therapeutics in Massachusetts, which funded the study.
